International journal of molecular sciences

Gene Editing Using CRISPR-Cpf1 Controlled by a Single Two-Way Promoter

Updated

Abstract

A compact adeno-associated virus (AAV) vector enables the co-expression of AsCpf1 and multiple CRISPR RNAs.

  • The single bidirectional H1 promoter supported indel formation on par with traditional dual-promoter systems.
  • This approach allows for scalable genome editing across single, dual, and triple target configurations.
  • Successful genome editing was achieved both in laboratory settings and in living organisms following AAV delivery.
  • The engineered AAV vector platform may enhance the feasibility of CRISPR-based therapies.

Simplified

Funding

Competing interests

Y.H.S. and G.-N.K. have registered a patent titled, “Single Adeno-associated virus (AAV) vector expressing CRISPR-Cpf1 using a bidirectional H1 promoter,” to the Korean Intellectual Property Office. The authors declare no conflicts of interest.
PubMed

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